Tackling Rare Diseases in India

Source: The post “Tackling Rare Diseases in India” has been created based on “Tackling Rare Diseases in India” published in “The Hindu” on 29th September 2026.

UPSC Syllabus: GS-3- Indian Economy

Context: Rare diseases individually affect a small number of people, but collectively affect hundreds of millions worldwide. India has reported 1,004 rare genetic disorders. The small market size has historically discouraged pharmaceutical companies from developing such medicines, leading to the term “orphan drugs.”

About Rare Disease

1. The World Health Organization (WHO) defines rare diseases as debilitating, lifelong conditions that affect one or fewer persons per 1,000.
2. Some commonly recognised rare diseases include Haemophilia, Pompe disease, Thalassemia, Sickle-cell Anaemia, and Gaucher’s disease.

Status of Rare Disease in India

1. In India, rare diseases are categorised into three groups based on the nature and complexity of available treatment options.
   1. Group 1 includes diseases that can be treated with a one-time curative procedure.
   2. Group 2 diseases require long-term or lifelong treatment which are relatively less costly and have shown documented benefits, but patients need regular check-ups.
   3. Group 3 diseases are those for which effective treatments are available, but they are expensive and must often continue lifelong. There are challenges in selecting the right beneficiaries for these treatments.
2. It is estimated that around 6%–8% of India’s population lives with a rare disease, translating to approximately 8.4 – 10 crore people.
3. Currently, 63 rare diseases are included under the National Policy for Rare Diseases, which was recommended by the Central Technical Committee for Rare Diseases (CTCRD).
4. Therapies are available for less than 5% of rare diseases, leading to less than 1 in 10 patients receiving disease-specific care.
5. Many medicines and therapies for rare diseases are patented, which makes them very expensive.
6. The market for these drugs is small and the development costs are also high, so pharmaceutical companies often don’t find it profitable to produce them, pushing up prices. Therefore, these drugs are called ‘orphan drugs’

Challenges

1. Limited commercial incentive: The small patient population reduces the incentive for pharmaceutical companies to invest in research and development of orphan drugs.
2. High cost of medicines: Rare-disease medicines can cost around ₹1 crore or more per patient annually, making them inaccessible without government support or insurance.
3. Insufficient financial support: Although the Government of India provides support of up to ₹50 lakh, this may not cover the total treatment cost, forcing families to depend on crowdfunding.
4. Limited availability of drugs: Existing rare-disease drugs address only around 5% of rare diseases and associated conditions, leaving most patients without suitable treatment.
5. Difficulty in clinical trials: Identifying adequate patient cohorts and determining suitable clinical endpoints are major challenges in developing orphan drugs.
6. Limited participation of Indian patients: Despite India’s large patient population, Indian patients are generally underrepresented in international orphan-drug clinical trials.
7. Small-scale manufacturing: After approval, producing and distributing medicines in small volumes at affordable prices while maintaining quality remains difficult.
8. Regulatory challenges: Greater transparency and clearer guidance for drug developers and sponsors can improve the regulatory approval process.
9. Access versus benefit-sharing: Patients participating in research should be able to access the resulting medicines. Developing expensive drugs that remain unaffordable locally limits the social benefit of such research.
10. Global competition: Countries such as the U.S.A and China are investing heavily in rare-disease drug development, creating both competition and opportunities for international collaboration.

India’s Opportunities

1. Large patient pool: India’s population provides a potentially large cohort for clinical trials. Certain communities also have higher prevalence of particular genetic disorders because of endogamy.
2. Patient advocacy networks: Patient groups and disease-specific registries can help identify suitable participants for clinical research.
3. Unique genetic diversity: Indian and diaspora populations possess unique genetic mutations, providing opportunities for research into rare diseases.
4. Strong pharmaceutical manufacturing base: India has established capabilities in generics, biosimilars and newer therapies such as CAR-T, and can manufacture medicines at relatively low cost.

Way Forward

1. Promote global–Indian collaboration: Encourage multinational pharmaceutical companies to establish rare-disease drug-development centres in India in collaboration with Indian organisations and patient groups.
2. Increase Indian participation in trials: Integrate Indian patients into international clinical trials while ensuring that participating communities can benefit from the resulting medicines.
3. Adopt benefit-sharing: Drug-development benefits should be shared fairly and equitably, including affordable access to medicines in India and the Global South.
4. Streamline regulation: Develop clearer regulatory guidance and reduce unnecessary duplication while maintaining bioequivalence, safety, toxicity, efficacy, pharmacovigilance and manufacturing standards.
5. Provide targeted incentives: Use production-linked incentives, tax incentives and investment support to encourage domestic orphan-drug manufacturing.
6. Support technology transfer: Facilitate technology transfer and process development through collaboration between pharmaceutical companies and national laboratories.
7. Use Advanced Market Commitments: The government can commit to purchasing a specified quantity at an agreed price, thereby providing manufacturers with predictable demand.
8. Strengthen public procurement and distribution: Medicines can be distributed through Centres of Excellence for Rare Diseases, CGHS and Pradhan Mantri Bhartiya Jan Aushadhi Kendras.
9. Promote exports: Incentives can encourage Indian manufacturers to supply affordable rare-disease medicines to low- and middle-income countries.
10. De-risk rather than merely subsidise: Government policy should reduce the initial financial risks sufficiently to make orphan-drug manufacturing commercially viable, rather than simply subsidising companies.

Conclusion: India already possesses the manufacturing capacity, large patient pool, scientific potential and pharmaceutical expertise required to enter the rare-disease drug sector. A combination of affordable access, patient participation, streamlined regulation, targeted incentives, government procurement and international collaboration can help India move from being the “pharmacy of the world” to becoming a significant producer of accessible rare-disease medicines.

Question: Rare diseases pose challenges of affordability, accessibility and limited research. Discuss how India can leverage its pharmaceutical capabilities to develop a globally competitive rare-disease drug industry.

Source: The Hindu